Eteplirsen (Exondys 51 – Sarepta), an antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have a mutation of the dystrophin gene that is amenable to exon 51 skipping. It is the first drug to be approved for treatment of DMD.

The FDA has approved datopotamab deruxtecan (Datroway – Daiichi Sankyo/AstraZeneca), a trophoblast cell-surface antigen-2 (Trop-2)-directed antibody and topoisomerase inhibitor conjugate, for treatment of adults with unresectable or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer who received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.

Alemtuzumab (Campath), a genetically engineered, humanized monoclonal antibody currently approved to treat B-cell chronic lymphocytic leukemia (BCLL) and used off-label for induction therapy in solid organ transplants, is now also being tried off-label for treatment of relapsing multiple sclerosis (MS).

The drugs of choice for treatment of fungal infections are listed in the table that begins on page 62. Some of the indications and dosages recommended here have not been approved by the FDA. More detailed guidelines for some of these infections are available online from the Infectious Diseases Society of America (www.idsociety.org).

Drugs for gout are used to reduce the pain and inflammation of acute flares, decrease the frequency of exacerbations, and lower serum urate levels to prevent recurrent flares, development of tophi, and joint damage.

Gemtuzumab ozogamicin, a monoclonal antibody bound to a cytotoxic antibiotic, has been approved by the FDA for treatment of patients with CD33-positive acute myeloid leukemia (AML) in first relapse who are more than 60 years old and may not be able to tolerate cytotoxic chemotherapy

The FDA has approved macitentan (ma" si ten' tan; Opsumit – Actelion), for oral treatment of pulmonary arterial hypertension (PAH). Macitentan is the second nonselective endothelin receptor antagonist approved for PAH. It is a derivative of bosentan (Tracleer), which is also manufactured by Actelion, and is scheduled to become available generically in 2015. Riociguat (Adempas), another new drug for this indication, will be reviewed in a future issue. Volibris

The FDA has approved obiltoxaximab (Anthim – Elusys Therapeutics), a toxin-targeting monoclonal antibody, for treatment of inhalational anthrax in combination with antibacterial drugs and prophylaxis of inhalational anthrax when other therapies are unavailable or inappropriate. Raxibacumab, another monoclonal antibody, was approved earlier for the same indication. Like raxibacumab, obiltoxaximab is being manufactured only for the CDC national stockpile and is not available for commercial sale.

The investigational monoclonal antibodies casirivimab and imdevimab (REGEN-COV – Regeneron) have been available in the US under an Emergency Use Authorization (EUA) since late 2020 for use together to treat mild to moderate COVID-19 in persons ≥12 years old who weigh ≥40 kg and are at high risk of progression to severe disease or hospitalization. The FDA has now expanded this EUA to allow use of the antibodies together for post-exposure prophylaxis of COVID-19 in such persons, if they are not fully vaccinated against COVID-19 or are unlikely to have an adequate immune response...

Tarlatamab-dlle (Imdelltra – Amgen), a first-in-class bispecific delta-like ligand 3 (DLL3)-directed CD3 T-cell engager, has received accelerated approval from the FDA for treatment of extensive-stage small cell lung cancer (SCLC) in adults who had disease progression on or after platinum-based chemotherapy. It is the first bispecific DLL3-directed CD3 T-cell engager to be approved in the US for this indication. Most patients with SCLC have a response to initial treatment, but progression generally occurs within a few months and overall survival is usually less than 8 months....